Sonidegib: Novartis Skin Cancer Drug Gets FDA Approval

Novartis’s Odomzo (sonidegib) has been approved by the US Food and Drug Administration (FDA). Approval was for treatment of adults with locally advanced basal cell carcinoma (laBCC) recurring after surgery or radiation therapy, and for individuals who are not good candidates for surgery or radiation therapy.

The FDA’s OK was based on demonstration of an objective response rate (ORR) in an international, multi-center, double-blind, randomized, two-arm, non-comparative trial.

Novartis Oncology President Bruno Strigini said in a statement:

“The FDA approval of Odomzo offers a new and non-invasive treatment option for a potentially devastating disease that is hard to treat and can be disfiguring. Odomzo is an important addition to our growing portfolio of targeted treatments for advanced skin cancers and underscores our commitment to developing and bringing to market new options for patients.”

Sonidegib, or erismodegib, also known as LDE225 and marketed as Odomzo, is a Hedgehog signaling pathway inhibitor (via smoothened antagonism) being developed as an anticancer agent by Novartis. It has also been investigated as a potential treatment for:

Pancreatic cancer
Breast cancer
Small cell lung cancer
Medulloblastoma
Advanced solid tumours
Chronic myeloid leukaemia
Myelofibrosis and essential thrombocythaemia

Odomzo was approved in Switzerland for the treatment of advanced BCC that is not amenable to curative surgery or radiotherapy on June 30, 2015. The CHMP granted a positive opinion on June 25, 2015.

Patients with laBCC treated with Odomzo 200 mg were followed for a minimum of 12 months, unless discontinued earlier. The ORR was 58% (95% confidence interval: 45, 70), consisting of 5% (n=3) complete responses (CR) and 53% (n=35) partial responses (PR).

A pre-specified sensitivity analysis using an alternative definition for CR, defined as at least a PR according to MRI and/or photography and no evidence of tumor on biopsy of residual lesion, yielded a CR rate of 20%.

Among the 38 patients with an objective response, 31 patients (82%) have ongoing responses ranging from at least 1.9 to 18.6 months and the median duration of response has not been reached.

Basal-cell carcinoma or basal-cell cancer (BCC), a skin cancer, is one of the most common cancers in the United States. It rarely metastasizes or kills. Since it can cause significant destruction and disfigurement by invading surrounding tissues, however, it is still considered malignant.

Worldwide incidence of BCC is rising by 10% each year due to factors such as an aging population and increased ultraviolet exposure. Incidence rates are estimated to be between 0.003% and 0.55% worldwide.

Illustration: S. Gschmeissner, K. Hodivala-Dilke & M. Stone, Wellcome Images